Precise plant genome editing using base editors and prime editors
The development of CRISPR–Cas systems has sparked a genome editing revolution in
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
[HTML][HTML] Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy
CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats-associated protein
9) shows the opportunity to treat a diverse array of untreated various genetic and …
9) shows the opportunity to treat a diverse array of untreated various genetic and …
[HTML][HTML] Fanzor is a eukaryotic programmable RNA-guided endonuclease
RNA-guided systems, which use complementarity between a guide RNA and target nucleic
acid sequences for recognition of genetic elements, have a central role in biological …
acid sequences for recognition of genetic elements, have a central role in biological …
Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing
AV Anzalone, XD Gao, CJ Podracky, AT Nelson… - Nature …, 2022 - nature.com
The targeted deletion, replacement, integration or inversion of genomic sequences could be
used to study or treat human genetic diseases, but existing methods typically require double …
used to study or treat human genetic diseases, but existing methods typically require double …
[HTML][HTML] Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety
advantages over nucleic acid delivery approaches. We report the development and …
advantages over nucleic acid delivery approaches. We report the development and …
Engineered pegRNAs improve prime editing efficiency
JW Nelson, PB Randolph, SP Shen, KA Everette… - Nature …, 2022 - nature.com
Prime editing enables the installation of virtually any combination of point mutations, small
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …
insertions or small deletions in the DNA of living cells. A prime editing guide RNA (pegRNA) …
[HTML][HTML] Enhanced prime editing systems by manipulating cellular determinants of editing outcomes
While prime editing enables precise sequence changes in DNA, cellular determinants of
prime editing remain poorly understood. Using pooled CRISPRi screens, we discovered that …
prime editing remain poorly understood. Using pooled CRISPRi screens, we discovered that …
[HTML][HTML] Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice
D Reichart, GA Newby, H Wakimoto, M Lun… - Nature medicine, 2023 - nature.com
Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
K Musunuru, AC Chadwick, T Mizoguchi, SP Garcia… - Nature, 2021 - nature.com
Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …
base editors 4, 5, have the potential to permanently modify disease-causing genes in …
[HTML][HTML] Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity
Cytosine base editors (CBEs) are larger and can suffer from higher off-target activity or lower
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …
on-target editing efficiency than current adenine base editors (ABEs). To develop a CBE that …